This Phase 2b/3, randomized, double-blind, placebo-controlled, 2-part study will evaluate the efficacy, safety and tolerability of different dose regimens of SPG601 in adult male participants with Fragile X syndrome.
Adult males aged 18 to 45 years, inclusive
Diagnosis of Fragile X as confirmed with genetic testing
Patient must have caregiver
Must be in good health with no significant medical history
Any physical or psychological condition that prohibits study completion
Uncontrolled seizures or history of epilepsy with a seizure in the past 6 months.
Auditory or visual impairments that cannot be corrected
History of suicidal behavior or suicidal ideation
Parallel
SPG601
Drugsynthetic small molecule
Placebo
DrugPlacebo
Phase 2b: Change from baseline in Total Cognition Composite Change Sensitive Score from the NIH-TCB
The NIH Toolbox (NIH-TCB) Total Cognition Composite Change-Sensitive Score (CSS) is used to measure cognitive change over time. Higher scores indicate higher level of functioning. The scores typically span across the developmental range, often extending well above and below the 500-point mark depending on the age and cognitive ability of the individual.
Phase 2b: Change from baseline in EEG resting state relative power bands during rest
Resting-state EEG relative power changes from baseline are characterized by significant shifts in power bands such as gamma, alpha, theta and beta bands
Phase 3:Change from baseline in Total Cognition Composite score from the NIH-TCB
The NIH Toolbox (NIH-TCB) Total Cognition Composite Change-Sensitive Score (CSS) is used to measure cognitive change over time. It is calculated by combining the Crystallized Composite (reading/vocabulary) and Fluid Composite (memory/executive function/speed) scores. Higher scores indicate higher level of functioning.
Phase 2b: Change from baseline in Fluid Reasoning Composite Change Sensitive Scores from the NIH-TCB
The Fluid Cognition Composite (FCC) from the NIH Toolbox Cognition Battery (NIHTB-CB) is designed to measure changes to neurological functioning. It combines scores from five tests (Dimensional Change Card Sort, Flanker, List Sorting, Pattern Comparison, and Picture Sequence Memory) to provide a measure of executive function, memory, and processing speed. Higher scores indicate higher level of functioning. Superior ability is 130, average is approx. 100 and less than 70 is impairment.
Phase 2b: Change from baseline in Flanker Scores from the NIH-TCB
Flanker Inhibitory control will assess inhibitory control and attention through asking participant to focus on one stimuli will not focusing on any stimuli surrounding it. A higher score indicates better performance
Phase 2b: Incidence, nature, and severity of adverse events (AEs) and serious adverse events (SAEs)
Incidence, nature, and severity of treatment emergent adverse events (TEAEs) and serious adverse events (SAEs)
Phase 2b: Change from baseline in Columbia-Suicide Severity Rating Scale (C-SSRS) throughout the study
Prospective suicidality assessment is performed using the Columbia-Suicide Severity Rating Scale (C-SSRS), a questionnaire to evaluate suicidal ideation and behavior. Answer "yes" on item 4 or 5 of the Suicidal Ideation section or "yes" on any item of the Suicidal Behavior section is considered positive. Range is 2-25
This trial is not recruiting at the moment. You can still explore other options:
